Rocket Pharmaceuticals unveiled a strategic re-org and pipeline prioritization, aimed at focusing resources on its adeno-associated virus (AAV) cardiovascular platform.
As part of the initiative, Rocket will implement a workforce reduction of approximately 30%, which the company says will reduce its 12-month operating expenses by nearly 25%.
The pipeline restructuring will direct resources towards Rocket's AAV cardiovascular platform, comprised of clinical programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy and BAG3-associated dilated cardiomyopathy. Rocket’s phase 2 Danon disease trial for gene therapy RP-A50 was placed on clinical hold in May after a patient, who later passed away after an acute systemic infection, experienced a serious adverse event.
The re-org will also enable the company to resolve the CMC deficiencies noted in the CRL that Rocket received for Kresladi, a lentiviral vector-based gene therapy to treat severe leukocyte adhesion deficiency-1, in June 2024.
Kresladi’s approval would grant Rocket a priority review voucher, which could be sold for additional cash. Excluding this possibility, Rocket says its existing cash resources will fund operations into the second quarter of 2027.
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